SynthesisEuropean archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery2023
Sirolimus treatment for paediatric head and neck lymphatic malformations: a systematic review.
Synthesis in European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 24 citations in OpenAlex.
- Therapeutic approaches to sinonasal NUT carcinoma: a systematic review.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2024Pooled it
- Safety of sirolimus in pediatric vascular anomalies: a BKMR-PopPK exposure-response framework for individualized risk and dosing.World journal of pediatrics : WJP · 2026Article
- Multidimensional outcomes of initial treatment strategies for pediatric head and neck lymphatic malformations.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026Article
- Sirolimus Therapy and Dyslipidemia in Neonates and Infants: A Retrospective Cohort and Pharmacovigilance Analysis.Paediatric drugs · 2026Article
- Targeted Therapies in Infantile Hemangiomas and Vascular Malformations: From β-Blockers to PI3K/AKT/mTOR Inhibitors.Journal of cellular and molecular medicine · 2026Review
- Retrospective evaluation of sirolimus (mTOR blocker) for lymphatic malformations presenting in the neonatal period: a single-center experience.Frontiers in pediatrics · 2026Article
- Emergency sclerotherapy for giant lymphatic malformation of the head and neck with airway obstruction in neonates.Frontiers in pediatrics · 2026Article
- Pediatric Lymphangioma of the Tongue, From Diagnosis to Surgery: A Case Report.Case reports in dentistry · 2026Article
- Glass-blowing, brass-playing, and other laryngocele etiologies: Fact or fiction? A systematic literature review.World journal of otorhinolaryngology - head and neck surgery · 2025Review
- Ultrasound-guided sclerotherapy of pingyangmycin for periorbital lymphatic malformations.BMC ophthalmology · 2025Article
- Treatments and Outcomes of Pediatric Head and Neck Lymphatic Malformations: A 20-Year Single Institution Experience.Otolaryngology--head and neck surgery : official journal of American Academy of Otolaryngology-Head and Neck Surgery · 2025Article
- Multidisciplinary therapeutic strategy with appropriate timing and modalities for treating cervicofacial lymphatic malformations in children.Pediatric surgery international · 2024Article
- Article
- A Veiled Lymphatic Malformation: Stridor in a Child.Cureus · 2024Article
- A multi-step approach to overcome challenges in the management of head and neck lymphatic malformations, and response to treatment.Orphanet journal of rare diseases · 2024Article
- Exploring New Drug Repurposing Opportunities for MEK Inhibitors in RASopathies: A Comprehensive Review of Safety, Efficacy, and Future Perspectives of Trametinib and Selumetinib.Life (Basel, Switzerland) · 2024Review
- Successful Management of Retropharyngeal Lymphangioma with Stridor Using Sirolimus: A Case Report.Indian journal of otolaryngology and head and neck surgery : official publication of the Association of Otolaryngologists of India · 2024Article
- Therapy with sirolimus in vascular anomalies: the experience of two Italian centers on 14 pediatric patients.Frontiers in pediatrics · 2024Article
- Repurposable Drugs for Immunotherapy and Strategies to Find Candidate Drugs.Pharmaceutics · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThis PRISMA-compliant systematic review aimed to assess risks and benefits of sirolimus treatment for paediatric lymphatic malformations by focusing not only on treatment efficacy but also on possible treatment-related adverse events, and treatment combinations with other techniques.
methodsSearch criteria were applied to MEDLINE, Embase, Web of Science, Scopus, Cochrane Library, and ClinicalTrials.gov databases and included all studies published up to March 2022 reporting paediatric lymphatic malformations treated with sirolimus. We selected all original studies that included treatment outcomes. After the removal of duplicates, selection of abstracts and full-text articles, and quality assessment, we reviewed eligible articles for patient demographics, lymphatic malformation type, size or stage, site, clinical response rates, sirolimus administration route and dose, related adverse events, follow-up time, and concurrent treatments.
resultsAmong 153 unique citations, 19 studies were considered eligible, with reported treatment data for 97 paediatric patients. Most studies (n = 9) were case reports. Clinical response was described for 89 patients, in whom 94 mild-to-moderate adverse events were reported. The most frequently administered treatment regimen was oral sirolimus 0.8 mg/m
conclusionDespite promising results for sirolimus treatment in lymphatic malformation, the efficacy and safety profile of remains unclear due to the lack of high-quality studies. Systematic reporting of known side effects, especially in younger children, should assist clinicians in minimising treatment-associated risks. At the same time, we advocate for prospective multicentre studies with minimum reporting standards to facilitate improved candidate selection.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.